Heart failure & cardiomyopathies
Early Access to Tafamidis for Patients With Transthyretin Amyloid Cardiomyopathy
Authors' conclusion
In an inclusive patient cohort receiving early access to tafamidis, safety findings were consistent with those reported from other trials and real-world studies.
Study context
- Population
- Patients with ATTR-CM who had not taken part in the phase 3 study, 2018–2023
- Participants
- 1,476 patients
- Outcome
- Safety, mortality and hospitalization
Methodological detailsnew independent cohort of the phase 3 long-term extension study, early access, minimal enrollment criteria
- New independent cohort of the phase 3 long-term extension study
- Early access
- Minimal enrollment criteria
Main finding
As reported in the abstract
All-cause and CV mortality and hospitalization (Kaplan-Meier, over study period) · median follow-up 19 months
- All-cause mortality
- 23.4%
- Cardiovascular (CV)-related mortality
- 13.8%
- All-cause hospitalization
- 43.3%
- CV-related hospitalization
- 26.5%
- Total annual CV-related hospitalization rate
- 0.26
Observational data: associations, not effects.
Safety
Treatment-related adverse events
- Treatment-related AEs
- 7.6%
- Serious treatment-related AEs
- 0.6%
- Treatment-related AEs leading to discontinuation
- 0.6%
- No new safety signals identified
Original publication
Early Access to Tafamidis for Patients With Transthyretin Amyloid Cardiomyopathy. JACC Adv. Published 10 September 2025. doi:10.
Composed by AI from the published abstract and checked against it. How Briefs are made →